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Non-cytotoxic systemic treatment in malignant peripheral nerve sheath tumors (MPNST): A systematic review from bench
Enrico Martin1, Nayan Lamba2, Uta E Flucke3
1Department of Plastic and Reconstructive Surgery, University Medical Center, Utrecht, The Netherlands.
Background:
Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive soft tissue sarcomas. Once metastasized, prognosis is poor despite regular treatment with conventional cytotoxic drugs. This study reviews the preclinical and clinical results of non-cytotoxic systemic therapy in MPNST.
Methods:
A systematic search was performed in PubMed and Embase databases according to the PRISMA guidelines. Search terms related to 'MPNST', 'targeted therapy', 'immunotherapy', and 'viral therapy' were used. Only in vivo studies and clinical trials were included. Clinicaltrials.gov was also searched for any ongoing trials including MPNST patients. Qualitative synthesis was performed on all studies stratifying per target: membrane, cytoplasmic, nuclear, immunotherapy and oncolytic viruses, and other. In vivo studies were assessed for treatment effect on tumor growth (low/intermediate/high), survival, and metastases. Clinical trials were assessed on response rate, progression-free survival, and overall survival.
Results:
After full-text screening, 60 in vivo studies and 19 clinical trials were included. A total of 13 trials are ongoing and unpublished. The included trials displayed relatively poor response rates thus far, with patients achieving stable disease at best. Inhibiting cytoplasmic targets most commonly yielded high treatment effect, predominantly after mTOR inhibition. Oncolytic viruses and angiogenesis inhibition also demonstrate intermediate to high effect. Therapies including a combination of drugs were most effective in controlling tumor growth. Several ongoing trials investigate potentially promising pathways, while others have yet to be established.
Conclusion:
Targeting the PI3K/Akt/mTOR pathway seems most promising in the treatment of MPNSTs. Oncolytic viruses and angiogenesis inhibition represent emerging therapies that require further study. Combinations of targeted therapies are most likely key to maximize treatment effect.
Insights
Non-cytotoxic therapies show promise for malignant peripheral nerve sheath tumors (MPNSTs). Targeting the PI3K/Akt/mTOR pathway and using oncolytic viruses or angiogenesis inhibitors are key areas for future MPNST treatment.
Area of Science:
- Oncology
- Pharmacology
Background:
- Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive soft tissue sarcomas with poor prognosis post-metastasis.
- Conventional cytotoxic drugs offer limited efficacy in treating advanced MPNSTs.
Purpose of the Study:
- To review preclinical and clinical outcomes of non-cytotoxic systemic therapies for MPNST.
- To identify promising therapeutic targets and strategies for MPNST treatment.
Main Methods:
- Systematic literature search of PubMed and Embase databases (PRISMA guidelines).
- Inclusion of in vivo studies and clinical trials focusing on targeted therapy, immunotherapy, and viral therapy for MPNST.
- Qualitative synthesis of studies based on therapeutic targets and assessment of treatment effects on tumor growth, survival, and clinical outcomes.
Main Results:
- 60 in vivo studies and 19 clinical trials were included; 13 trials are ongoing.
- Inhibiting cytoplasmic targets, particularly via mTOR inhibition, demonstrated high treatment effects.
- Oncolytic viruses and angiogenesis inhibitors showed intermediate to high efficacy; combination therapies were most effective.
Conclusions:
- The PI3K/Akt/mTOR pathway is a highly promising target for MPNST treatment.
- Oncolytic viruses and angiogenesis inhibition are emerging therapies requiring further investigation.
- Combination targeted therapies are crucial for maximizing treatment efficacy in MPNST.
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